A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
Public ClinicalTrials.gov record NCT06555419. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open Label Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP). The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics, Inc. It consists of a catheter, which is a flexible tube, connected to a port which is placed under the skin. Alcyone Therapeutics, Inc. has an ongoing study called PIERRE to test the ThecaFlex DRx system. Participants with spinal muscular atrophy (SMA) in the PIERRE study may be enrolled in the PIERRE-PK study. The main goal of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are: * What is the highest amount of nusinersen found in the blood after dosing? * How much nusinersen is found in the blood over the first 24 hours after dosing? The PIERRE-PK study will be done as follows: * Participants will be screened to check if they can join the study. The screening period will be up to 30 days for this study and may overlap with the PIERRE study. * Participants will join 1 of 2 groups based on their ongoing nusinersen treatment. One group will be receiving 12 mg doses of nusinersen. The other group will be receiving 28 mg doses of nusinersen. * Participants will first receive a dose of nusinersen by lumbar puncture. * The ThecaFlex DRx system will be implanted after the lumbar puncture, as part of the PIERRE study. * Participants will receive a dose of nusinersen by the ThecaFlex DRx system, as part of the PIERRE study. This dose will be a regular maintenance dose that happens about 4 months after their first dose in this study. * Researchers will take blood samples before and after each dose. The last blood sample will be taken 24 hours after the dose. * The total study duration for each participant in the PIERRE-PK study will be approximately 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.
Study identification
- NCT ID
- NCT06555419
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1
- Enrollment
- 78 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 3 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 15, 2025
- Primary completion
- Sep 29, 2027
- Completion
- Sep 29, 2027
- Last update posted
- Sep 2, 2026
2025 – 2027
United States locations
- U.S. sites
- 12
- U.S. states
- 9
- U.S. cities
- 12
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| St. Joseph's Hospital and Medical Center - Barrow Neurological Institute | Phoenix | Arizona | 85013 | Recruiting |
| Children's Hospital of Orange County | Orange | California | 92868 | Recruiting |
| Stanford University Medical Center | Department of Neurology_Palo Alto | Palo Alto | California | 94304 | Recruiting |
| Nemours Children's Hospital, Orlando | Orlando | Florida | 32827 | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611-2605 | Recruiting |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Recruiting |
| Helen DeVos Children's Hospita | Grand Rapids | Michigan | 49503 | Recruiting |
| Milton S. Hershey Medical Center | Pennsylvania State University_Hershey | Hershey | Pennsylvania | 17033 | Recruiting |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Recruiting |
| Texas Childrens Hospital Houston | Houston | Texas | 77030 | Recruiting |
| Stacey Hall Developmental Pediatrics | Charlottesville | Virginia | 22903 | Recruiting |
| Children's Hospital of the King's Daughters_Norfolk | Norfolk | Virginia | 23507 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 9 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06555419, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 2, 2026 · Synced Sep 8, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06555419 live on ClinicalTrials.gov.